Duchenne MD Drug Could Be Effective in Fighting SMA

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In this video from Wochit News, we find out a little information about a drug which has shown promise in mouse models in regressing spinal muscular atrophy (SMA).

Clinical development for SMA treatments reviewed at an annual conference. Find out more. 

The drug Pip6a-PMO, is designed for patients with Duchenne muscular dystrophy but showed promise in disease regression in mice with SMA. The researchers will be undertaking clinical trials in patients affected by SMA next year and hope that the drug will be successful and can be used for other similar diseases. Find out more about this drug and the proposed new delivery system for the treatment here.

Update on stage 2 clinical trial of nusinersen in SMA infants presented by Ionis Pharmaceuticals.

SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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